The present disclosure provides modified nucleosides, nucleotides, and nucleic acids, and methods of using them.

Modified nucleosides, nucleotides, and nucleic acids, and uses thereof

Provided are compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the use of modified messenger RNAs, and are useful to treat or prevent diseases, disorders or conditions, or to improve a subject's heath or wellbeing.

Engineered nucleic acids and methods of use thereof

The present invention relates to the composition of polynucleotides, primary transcripts, and mmRNA molecules, and methods for their preparation, manufacture, and therapeutic use.

Modified polynucleotides for the production of biologics and proteins associated with human disease

Provided are formulations, compositions and methods for delivering biological moieties such as modified nucleic acids into cells to modulate protein expression. Such compositions and methods include the delivery of biological moieties, and are useful for production of proteins.

Delivery and formulation of engineered nucleic acids

The disclosure relates to compositions and methods for the preparation, manufacture and therapeutic use of polynucleotides. More specifically, the disclosure provides compositions, methods, processes, kits and devices for the design, preparation, manufacture and/or formulation of modified mRNA (mmRNA) molecules from primary transcripts. Methods of using mmRNA for treating conditions or diseases are also disclosed.

Modified polynucleotides for the production of secreted proteins

The present invention relates to the identification of new proteins comprising fibroblast growth factor 21 (FGF21) and other metabolic regulators, including variants thereof, known to improve metabolic profiles in subjects to whom they are administered. Also disclosed are methods for treating FGF21-associated disorders, GLP-1-associated disorders, and Exendin-4-associated disorders, including metabolic conditions.

Dual function proteins for treating metabolic disorders

Disclosed herein is pyrroline-carboxy-lysine (PCL), a pyrrolysine analogue, which is a natural, biosynthetically generated amino acid, and methods for biosynthetically generating PCL. Also disclosed herein are proteins, polypeptides and peptides that have PCL incorporated therein and methods for incorporating PCL into such proteins, polypeptides and peptides. Also disclosed herein is the site-specific derivatization of proteins, polypeptides and peptides having PCL or pyrrolysine incorporated therein. Also disclosed herein is the crosslinking of proteins, polypeptides and peptides having PCL or pyrrolysine incorporated therein.

Biosynthetically generated pyrroline-carboxy-lysine and site specific protein modifications via chemical derivatization of pyrroline-carboxy-lysine and pyrrolysine residues

Erratum: Structure of DDn, the deazaflavin-dependent nitroreductase from mycobacterium tuberculosis involved in bioreductive activation of PA-824 (Structure (2012) 20 (101-112))

The present invention provides antibody drug conjugates, wherein an antibody or antibody fragment that specifically binds to human cKIT is linked to a drug moiety, optionally through a linker. The present invention further provides pharmaceutical compositions comprising the antibody drug conjugates; and methods of making and using such pharmaceutical compositions for ablating hematopoietic stem cells in a patient in need thereof.

Antibody drug conjugates for ablating hematopoietic stem cells

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Papers

Dual function proteins for treating metabolic disorders

Biosynthetically generated pyrroline-carboxy-lysine and site specific protein modifications via chemical derivatization of pyrroline-carboxy-lysine and pyrrolysine residues

Erratum: Structure of DDn, the deazaflavin-dependent nitroreductase from mycobacterium tuberculosis involved in bioreductive activation of PA-824 (Structure (2012) 20 (101-112))

Antibody drug conjugates for ablating hematopoietic stem cells